MUCOLIPIDOSIS TYPE IV
Clinical trials for MUCOLIPIDOSIS TYPE IV explained in plain language.
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Experimental gene therapy hopes to tackle rare childhood disease
Disease control ENROLLING_BY_INVITATIONThis early-stage study tests a gene therapy called AAV9.hMCOLN1co in one child with Mucolipidosis Type IV, a rare genetic disorder. The goal is to see if the treatment is safe and can help control the disease. Participants must be between 1.5 and 8 years old and have a confirmed …
Matched conditions: MUCOLIPIDOSIS TYPE IV
Phase: PHASE1 • Sponsor: The Children's Hospital of Zhejiang University School of Medicine • Aim: Disease control
Last updated May 15, 2026 11:54 UTC
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New study tracks rare disease MLIV to unlock its secrets
Knowledge-focused OngoingThis study looks back at medical records of 50 people with mucolipidosis type IV (MLIV) to describe how the disease progresses. Researchers want to know when children reach or lose developmental milestones, how their movement and vision change, and what brain scans show. The goal…
Matched conditions: MUCOLIPIDOSIS TYPE IV
Sponsor: Massachusetts General Hospital • Aim: Knowledge-focused
Last updated May 12, 2026 13:39 UTC